TASK ORDER AWARD. CHEMISTRY, MANUFACTURING AND CONTROLS AND RELATED SERVICES FOR DEVELOPMENT OF DRUG SUBSTANCES. 3-YEAR STABILITY STUDY EXTENSION ON THE PTH-IA CGMP BATCH

Investigators

Abstract

The National Center for Advancing Translational Sciences (NCATS), through its Therapeutics for Rare and Neglected Diseases (TRND) program, is collaborating with the Massachusetts General Hospital (MGH) to evaluate [Leu11,dTrp12,Trp23Tyr36]-PTHrP(7-36)NH2 (PTH-IA) peptide as a potential treatment for Jansen Metaphyseal Chondrodysplasia (JMC). JMC is a rare disease of skeletal development and mineral ion homeostasis. Therapeutical Development Branch (TDB) in NCATS is responsible for the late-stage pre-clinical development toward an Investigational New Drug (IND) application and clinical testing of the PTH-IA. One cGMP lot of PTH-IA acetate have been acquired by TDB to support the preclinical safety evaluation. The purpose of this contract is for TDB/NCATS to acquire an additional 150 GN (on net peptide basis) of cGMP PTH-IA acetate to allow for additional studies to be conducted, and to conduct a three year stability study of the cGMP batch. Under this task order, the contractor will be responsible for producing a 150 GN Current Good Manufacturing Practices (cGMP) lot of PTH-IA peptide, generate a CMC report in eCTD format, and conducting 3-year stability studies of PTH-IA peptide GMP batch (per ICH guidelines) using developed and validated stability-indicating analytical methods. PTH-IA peptide structure: H-Leu-Leu-His-Asp-Leu-dTrp-Lys-Ser-Ile-Gln-Asp-Leu-Arg-Arg-Arg-Phe-Trp-Leu-His-His-Leu-Ile-Ala-Glu-Ile-His-Thr-Ala-Glu-Tyr-NH2 or LLHDLXKSIQDLRRRFWLHHLIAEIHTAEY; X = dTrp The counter ion: acetate